Euro Roundup: EMA posts draft guidance on running clinical trials in public health emergencies

The European Medicines Agency (EMA) is seeking feedback on draft guidance about conducting clinical trials during public health emergencies (PHEs).

EMA’s guidance applies to trials of interventions that could help address the PHE and studies of drug candidates unrelated to the crisis. The agency said trials addressing other serious or life-threatening medical needs, and, where feasible, other ongoing or planned clinical development work can continue in a PHE, provided patient safety and data integrity can be maintained.

National authorities should prioritize assessments of clinical trials that are essential for understanding, mitigating, or addressing the emergency, EMA said. When necessary, authorities can place less urgent trials on hold to safeguard regulatory capacity.

EMA wants sponsors to assess the feasibility and proportionality of initiating new trials during a PHE, considering the available infrastructure and collaboration opportunities. The agency said coordination at national and European Union levels is important to avoid duplication and generate meaningful results. Small COVID-19 trials and single-country compassionate use programs failed to yield robust evidence.

The draft also covers changes to ongoing clinical trials. EMA said sponsors should make every effort to adapt ongoing studies to obtain relevant clinical trial results as fast as possible. Changing existing trials may be the most efficient way to generate data relevant to a PHE, leading EMA to suggest sponsors could adapt endpoints to the emergency or include a new cohort with a different indication.

Authorities would classify such changes as substantial modifications. Sponsors may also need to make other major adjustments, such as implementing remote consent or temporarily halting enrollment at some or all investigator sites. Authorities will expedite the assessment of substantial modifications that are a direct consequence of the PHE.

Another section of the draft provides detailed guidance on trial processes sponsors may change to adapt ongoing trials to PHEs. The section features points to consider when adapting informed consent, safety monitoring, investigational product management, and other processes to emergencies.

In exceptional cases, sponsors may make changes without waiting for approval. EMA wants to limit that practice to situations where immediate action is required, for example because patient safety or data integrity are at risk.

EMA is seeking feedback on the draft until 30 April.

Draft Guidance

Notified bodies warn MDR, IVDR revisions risk reducing regulatory scrutiny

Notified body association Team-NB has warned that proposed revisions to the medtech regulations could cut oversight to below the level provided by the old directives.

The EU established the Medical Device Regulation (MDR) and In Vitro Diagnostic Regulation (IVDR) to improve on the oversight mandated under the directives and thereby prevent the recurrence of scandals like the use of non-medical grade silicone in breast implants. Facing implementation challenges, the EU has proposed steps to reduce burdens, promote innovation, and enable earlier regulatory interactions.

Team-NB said it fully supports the objective of improving predictability, consistency, and transparency in conformity assessment. However, the association is concerned about planned reductions in oversight activities including quality management system audits, technical documentation sampling, and vigilance data reviews.

While reduced scrutiny may create short-term administrative relief, Team-NB warned it can undermine patient safety, weaken market trust, and require substantially more surveillance resources from competent authorities. The association said safeguarding patient safety should remain the primary objective, and reductions in oversight should only be considered where supported by solid data.

Team-NB also questioned the European Commission’s estimate that changes to conformity assessment will save €2.1 billion ($2.4 billion), arguing that even substantial reductions in notified body activity could not produce such savings without severely compromising oversight.

Other concerns center on the potential unintended consequences of discounting notified body fees for micro and small manufacturers. Most micro and small manufacturers placing devices on the EU market are based outside the region, Team-NB said. The association is worried that discounted fees will attract more non-EU companies, intensifying pressures on local medtech companies.

Although Team-NB has specific concerns, the association said notified bodies support many elements of the proposed revisions to MDR and IVDR. Efforts to enhance predictability, improve regulatory dialogue, support digitalization, and streamline innovative pathways are particularly welcome, Team-NB said.

Team-NB Paper

EMA expands AI-enabled search tool to cover drug public assessment reports

EMA published the third major release of Scientific Explorer, expanding the artificial intelligence-enabled tool to include European public assessment reports (EPARs) for initial marketing authorization applications.

The tool is a search engine designed to help the European Medicines Regulatory Network’s scientific staff find information. Initially launched in 2024, the tool features a set of AI prompts that help EMA staff and other officials extract regulatory and scientific information from certain documents. The first two versions of the tool only searched scientific advice letters relating to human and veterinary medicines.

Version three adds the ability to search the EPARs that EMA publishes for every human or veterinary medicine application that has been granted or refused a marketing authorization. EPARs feature public information on a medicine, including how it was assessed by EMA, and reflect the scientific conclusions of the committee that reviewed the application.

EMA FAQs

Swissmedic revises rules for presenting undesirable effects and interactions

The Swiss Agency for Therapeutic Products (Swissmedic) has updated the requirements for presenting information about undesirable effects and interactions.

Swissmedic has overhauled the undesirable effects section of its guidance on product information for human drugs, including by clarifying the procedure for pooling safety data. The agency now specifies a tabular format to list the frequency of adverse effects.

Officials have reduced the level of detail concerning the requirements in the interactions section, shifting the focus to clinically relevant information. Companies now only need to give the ratios of the pharmacokinetic parameters for in vivo studies. Negative in vivo or in vitro data should only be described briefly. Swissmedic has also added support for using in silico data.

Other changes are intended to protect the environment, with Swissmedic adding a reference to ecotoxicological investigations to the preclinical data section and providing instructions on proper disposal.

Swissmedic Notice

EMA updates advice on out-of-specification cell- and tissue-based therapies

EMA has updated a question-and-answer document about the use of out-of-specification (OOS) batches of authorized cell- and tissue-based advanced therapy medicinal products.

The agency published the first version of the Q&A in 2019 to explain when OOS batches can be given to patients and describe the process for notifying authorities. EMA has retained most of the original advice in the updated Q&A but has completely rewritten its response to a question about how manufacturers and importers can notify its staff about OOS batches.

EMA expects companies to submit a periodic review of the OOS batches that have been administered. The periodic review should be filed as a quality defect report every six months and include an overview of OOS batches compared to in-specification batches. The goal is to verify the consistency of the process and the suitability of the specifications.

EMA Q&A

Other News:

Pharma trade groups have called on Ministers of Economy to ask the European Commission to stop the clock on implementation of the Urban Wastewater Treatment Directive’s extended producer responsibility scheme. EFPIA, Medicines for Europe, and other groups said urgent action is needed to avoid significantly harming companies operating in Europe. Press Release

In a joint announcement, the UK Space Agency, Medicines and Healthcare products Regulatory Agency (MHRA), and several other government authorities said they are working to develop a “supportive regulatory environment to space” in order to advance in-orbit manufacturing. Press Release

Euro Roundup: EMA posts draft guidance on running clinical trials in public health emergencies

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